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MedPage Today on MSN2d
Shipments of Duchenne Gene Therapy to Resume After FDA Review of Patient Death
Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver ...
Amazon S3 on MSN1d
The story of Sarepta's Duchenne gene therapy
The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The ...
Fierce Pharma3d
FDA takes U-turn on Sarepta's Elevidys, backing Duchenne gene therapy again in ambulatory patients
Monday, the FDA said Sarepta may resume treating ambulatory DMD patients with Elevidys. The recommendation comes just 10 days ...
Just The News1d
FDA allows distribution of muscular dystrophy drug again after public criticism
The agency’s now-reversed decision to halt distribution of a gene therapy for children suffering from muscular dystrophy ...
STAT6d
For many Duchenne families, halt to gene therapy is heartbreak upon heartbreak
As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...
STAT3d
FDA permits use of Sarepta Therapeutics’ Duchenne therapy in younger patients after short-lived halt
The FDA said it was clearing the way for Sarepta Therapeutics to resume shipments of its gene therapy for Duchenne muscular ...
FDA Suspends Muscular Dystrophy Drug After 3 Patient Deaths
The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...
Parents call on FDA for path forward after Duchenne's gene therapy pause | Morning in America
The Food and Drug Administration has paused distribution of the gene therapy Elevidys following the death of a third patient ...
Pharmabiz1d
Genethon gets UK MHRA nod to initiate pivotal phase 3 trial of GNT0004, a low-dose microdystrophin gene therapy for Duchenne muscular dystrophy
Genethon gets UK MHRA nod to initiate pivotal phase 3 trial of GNT0004, a low-dose microdystrophin gene therapy for Duchenne muscular dystrophy: Paris, France Wednesday, July 30, ...
BioPharma Dive10d
FDA asks Sarepta to stop shipping Duchenne gene therapy
The company refused the FDA’s request and will continue shipping its therapy, Elevidys, to Duchenne patients who can still ...
Sarepta Therapeutics crisis is huge blow to Duchenne families, company
Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...